This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).
Diagnosed with one of the following advanced mastocytosis diagnoses by Eligibility Committee
Aggressive Systemic Mastocytosis (ASM)
Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
Mast Cell Leukemia (MCL)
Persistent toxicity from previous therapy for AdvSM that has not resolved to ≤ Grade 1
Associated hematologic neoplasm requiring immediate antineoplastic therapy
Clinically significant cardiac disease
Known positivity for the FIP1L1 PDGFRA fusion. Patients with eosinophilia without detectable KIT D816V mutation must demonstrate lack of PDGFRA fusion mutation prior to enrollment
Single group
bezuclastinib
DrugBezuclastinib is administered as tablets to be taken orally, continuously in 28-day cycles.
Part I: Identify clinically active and tolerable exposures of bezuclastinib in patients with AdvSM
Part II: - Determine efficacy of bezuclastinib as measured by mIWG Objective Response Rate (ORR) - Confirm the exposure-response relationship of bezuclastinib
Pure Pathologic Response (PPR)
Months
Safety of CGT9486 as assessed by incidence of Adverse Events (AEs)
Incidence of AEs according to CTCAE version 5.0 or higher
To determine the effects of bezuclastinib on mutation allele burden.
Percentage change in KIT D816V
To determine the effects of bezuclastinib on serum tryptase.
Percentage change in Serum Tryptase
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